Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing.

ABSTRACT:

Background

Human induced pluripotent stem cells (iPSCs) play an important role in disease modeling and drug testing. However, the current methods are time-consuming and lack an isogenic control.

Objectives

This study sought to establish an efficient technology to generate human PSC-based disease models with isogenic control.

Methods

The ion channel genes KCNQ1 and KCNH2 with dominant negative mutations causing long QT syndrome types 1 and 2, respectively, were stably integrated into a safe harbor AAVS1 locus using zinc finger nuclease technology.

Results

Patch-clamp recording revealed that the edited iPSC-derived cardiomyocytes (iPSC-CMs) displayed characteristic long QT syndrome phenotype and significant prolongation of the action potential duration compared w

SUBMITTER: Wang Y 

PROVIDER: S-EPMC4149735 | biostudies-literature | 2014 Aug

REPOSITORIES: biostudies-literature